The Clinical Pharmacist's Role in Curative Gene Therapies: A Patient Journey Framework for Access, Safe Use, and Long-Term Monitoring - A Narrative Review

Background: The approval of curative gene therapies for haemophilia A and B, sickle cell disease, and spinal muscular atrophy has introduced a category of clinical intervention with no precedent in conventional pharmaceutical care. These therapies demand a level of pharmacist expertise spanning genomics, complex logistics, financial navigation, and decades-long safety surveillance that the current pharmacy workforce is not consistently prepared to provide. Objective: To develop a comprehensive, patient journey framework mapping clinical pharmacist responsibilities across all phases of curative gene therapy delivery, and to define the core competency domains required for effective pharmacist integration in this setting. Methods: A narrative review was conducted searching MEDLINE/PubMed, Scopus, Web of Science, Embase, and Google Scholar from January 2015 through December 2025, supplemented by regulatory documents, REMS communications, and professional society repositories. Evidence from clinical trials, cohort studies, regulatory guidance, and consensus statements was synthesized narratively across five sequential phases: candidate identification, access navigation, preparation, administration, and long-term monitoring. Results: Across all five phases, pharmacists fulfil critical and non-substitutable roles. These include neutralizing antibody screening and medication reconciliation at eligibility assessment; prior authorization, outcomes-based contract navigation, and chain-of-identity oversight during access and acquisition; busulfan therapeutic drug monitoring and bridging therapy optimization during conditioning; pre-medication protocol management and acute reaction response during administration; and corticosteroid taper monitoring, platform-specific toxicity surveillance, and FDA-mandated registry stewardship during long-term follow-up. Disease-specific analyses of haemophilia, sickle cell disease, and SMA illustrate how these responsibilities are shaped by platform biology, patient population, and product-specific safety obligations including Boxed Warnings for hepatotoxicity, insertional oncogenesis, and thrombotic microangiopathy. Five core pharmacist competency domains were identified: clinical genomics literacy, access and reimbursement management, operational and quality oversight, advanced toxicity monitoring and survivorship care, and patient and caregiver education. Conclusion: Clinical pharmacist integration is essential to the safe, equitable, and operationally sound delivery of curative gene therapies. Achieving this integration requires urgent investment in pharmacy education, residency training, and formal credentialing pathways commensurate with the complexity these therapies demand.